Doctors successfully cure baby with first-ever personalized gene editing

A team of doctors and scientists has made a medical breakthrough by successfully curing a rare genetic disease using the world's first personalized gene editing therapy, Engadget reports. The results of this groundbreaking treatment were published in The New England Journal of Medicine.

The patient was a baby named KJ, who was born with a deficiency of the enzyme CPS1. This rare condition has a high mortality rate — about 50% of babies die within the first week of life. Those who survive can face serious neurological impairments, developmental delays, and the need for a liver transplant.

The treatment team developed a personalized therapy based on CRISPR, a human DNA editing technology that works like molecular "scissors" that can precisely alter DNA, correcting disease-causing mutations.

This approach could be used in the future to treat other genetic diseases, such as sickle cell anemia, cystic fibrosis, and muscular dystrophy.